Jerry Mendell, M.D.

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Jerry Mendell, director of the Center for Gene Therapy at the Research Institute at Nationwide Children’s Hospital in Columbus, Ohio, was awarded an MDA translational research grant totaling $458,814 to develop bloodstream delivery of alpha-sarcoglycan genes to people with type 2D limb-girdle muscular dystrophy (LGMD2D). This form of LGMD arises from a deficiency of the alpha-sarcoglycan protein. The grant will allow Mendell and his colleagues to conduct the testing required by the U.S. Food and Drug Administration (FDA) before the agency can give permission for a clinical trial of bloodstream-delivered gene therapy in LGMD2D patients.